LEADERSHIP &
ADMINISTRATION
Executive Committee
Adeline L. Vanderver, MD
Children’s hospital of Philadelphia
Adeline Vanderver, MD, is an Attending Physician in the Division of Neurology at Children’s Hospital of Philadelphia (CHOP) and the Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics. She is also Program Director of the Leukodystrophy Center of Excellence at CHOP.
Dr. Vanderver graduated with a degree in medicine from Universite Catholique de Louvain in Brussels, Belgium. She completed her residency in pediatrics at Nemours/A.I. duPont Hospital for Children in Wilmington, DE, and Thomas Jefferson University in Philadelphia, PA. She then pursued a child neurology fellowship at Children's National Medical Center in Washington, DC, and a fellowship in biochemical genetics at the National Human Genome Research Institute/National Institutes of Health in Bethesda, MD.
Dr. Vanderver’s has helped clarify the molecular etiology over a dozen leukodystrophies and has contributed more than 100 peer reviewed manuscripts to the literature on leukodystrophies over the past decade. She has contributed to the development of new standards of care for children with leukodystrophies by advancing leukodystrophy gene discovery, creating new therapies, and supporting and advocating for patients and their families. In addition to providing scientific and administrative direction to the Global Leukodystrophy Initiative Clinical Trials Network, she has directed Project 4 and co-directed Project 1 and Project 3.
Florian Eichler, MD
Massachusetts General Hospital
Florian Eichler, MD, is the Director of the Leukodystrophy Service and the Center for Rare Neurological Diseases at Massachusetts General Hospital (MGH), as well as an Associate Professor of Neurology at and Harvard Medical School. His laboratory explores the relationship of mutant genes to specific biochemical defects and their contribution to neurodegeneration.
Dr. Eichler obtained his medical degree from the University of Vienna Medical School. After graduating from medical school in 1997, he entered residency in Pediatrics and pursued studies on cerebral blood flow and metabolism at the University of Vienna. In 1999 he won a scholarship to study in vivo MR spectroscopy in pediatric patients with metabolic and neurometabolic conditions at Johns Hopkins (Stipendium Metabolicum 1999) and joined the laboratory of Dr. Hugo Moser dedicated to peroxisomal disorders at the Kennedy Krieger Institute. Following his research fellowship at Johns Hopkins he underwent residency training in Child Neurology at the Massachusetts General Hospital (MGH). After completing residency in 2005 he joined the staff at MGH.
Dr. Eichler is the principal investigator of several NIH- funded studies on neurogenetic disorders, including a natural history study of Canavan Disease (NCT02851563), ALD (NCT03278899) as well as a gene therapy trial of adrenoleukodystrophy. He also serves as chair of the Rare Disease Think Tank at MGH and is founder of the international consortium ALD Connect, a patient powered research network. In addition to providing scientific and administrative direction to the Global Leukodystrophy Initiative Clinical Trials Network, he has co-directed Projects 1 and Project 2, and has contributed to Project 4.
S. Ali fatemi, MD, MBA
Kennedy Krieger Institute
S. Ali Fatemi, MD, is the Chief Medical Officer at Kennedy Krieger Institute, a pediatric neurologist and the director of the Division of Neurogenetics and the Moser Center for Leukodystrophies, and an investigator at the Hugo W. Moser Research Institute at Kennedy Krieger Institute. Dr. Fatemi is also an associate professor of neurology and pediatrics at Johns Hopkins University.
Dr. Fatemi graduated from the Medical University of Vienna in Austria. He completed a postdoctoral fellowship under the mentorship of Hugo W. Moser, during which he developed neuroimaging studies for leukodystrophies. He received clinical training at SUNY Downstate, and Massachusetts General Hospital and returned to Kennedy Krieger Institute in 2008 as a faculty member. In 2009, He received an NINDS K08 career development award to study developmental myelination and glial progenitor cell therapy.
His current research activities encompass both basic and clinical research, and he has served as site PI for several clinical trials. In addition to providing scientific and administrative direction to the Global Leukodystrophy Initiative Clinical Trials Network, he has co-directed Project 2, and has contributed to Project 1 and Project 4.
Program Manager
Omar S. Sherbini, MPH
research ADMINISTRATIVE Director
Children’s Hospital of Philadelphia
Omar Sherbini is the Administrative Director for the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) and also serves as an Administrative Director within the Leukodystrophy Center at Children’s Hospital of Philadelphia, where he oversees a team of clinical research coordinators, technicians, and scientists working on various biorepository and natural history studies.
Omar holds a Master of Public Health (MPH) degree from the Johns Hopkins Bloomberg School of Public Health, with a concentration in infectious diseases. He began his scientific career in the Institute for Cell Engineering at Johns Hopkins University, where he investigated stroke pathophysiology and neuroprotective agents in animal models.
Connect with Omar via LinkedIn at http://www.linkedin.com/in/omarsherbinimph.
Advocacy Committee
ERICA BARNES, MS, CCC-SLP
glia-ctn advocacy committee chair
EXECUTIVE DIRECTOR, MINNESOTA RARE DISEASE ADVISORY COUNCIL
Erica Barnes is the current GLIA-CTN Advocacy Committee Chair, and also serves as Executive Director of the Minnesota Rare Disease Advisory Council. In 2010, when Erica tragically lost her young daughter, Chloe, to metachromatic leukodystrophy (MLD), a rare disease, Erica found herself in the role of rare disease advocate. She has been active in the advocacy community for over a dozen years, championing policies related to timely diagnosis, appropriate care, and effective treatments.
Erica began her professional career as a speech-language pathologist in the healthcare setting. Following the death of her daughter, Chloe, in 2010 she co-founded Chloe’s Fight Rare Disease Foundation with her husband Philip. Through her advocacy work she has represented the National Organization for Rare Disorders (NORD) as a Minnesota state ambassador, has provided public testimony at the federal level in support of newborn screening, and frequently speaks at national conferences and contributes to publications focused on rare diseases.
Connect with Erica via LinkedIn at https://www.linkedin.com/in/ericadbarnes.
MELODY KISOR, MS
GLIA-CTN ADVOCACY COMMITTEE CO-CHAIR
DIRECTOR OF PATIENT ENGAGEMENT, CURE LBSL
Melody Kisor, MS proudly serves as the GLIA-CTN Advocacy Committee Co-Chair along with her esteemed colleague, Erica Barnes. She also leads the GLIA-CTN Patient Registry workgroup, and is an active member of the GLIA-CTN FDA Engagement workgroup.
The majority of Melody’s career has been spent in public health, including birth defects research/surveillance and perinatal health education. However, rare disease advocacy is her true calling. Melody earned a Master of Science in Biomedical Ethics with a concentration in maternal/child and genetic ethics from Albany Medical College. She also holds a BA in Interdisciplinary Health Studies from Hampshire College, with concurrent epidemiology coursework at the University of Massachusetts Amherst Graduate School of Public Health. Melody also holds a specialized certificate in Perinatal Health Education from the University of California San Diego (UCSD).
Melody first heard the word “leukodystrophy” when her oldest brother was diagnosed with an adult-onset form of unknown etiology (now known to be ALSP). She heard it again when a neighborhood child was diagnosed with LBSL. That connection brought her back into the leukodystrophy world, where she now uses her bioethics skills to amplify the rare patient/caregiver voice.
Connect with Melody via LinkedIn at https://www.linkedin.com/in/melodykisor.
SARAH STONEY, MSW, LCSW
GLIA-CTN ADVOCACY LIAISON
OUTPATIENT SUBSPECIALITY SOCIAL WORKER
CHILDREN'S HOSPITAL OF PHILADELPHIA
Sarah Stoney, MSW, LCSW is a social worker at the Children’s Hospital of Philadelphia with over a decade’s worth of experience working with patients with complex neurological conditions. During her time in the hospital’s Division of Neurology, she has worked with a diverse set of clinical groups, including our Mitochondrial Medicine Program, Leukodystrophy Center, Neuromuscular Program, Pediatric Multiple Sclerosis and Neuroinflammatory Disorders Clinic. She helps patients and their families navigate the complex medical system, assists with advocacy in schools and throughout the community, and provides ongoing support to families.
Sarah has cultivated relationships with many leaders within advocacy community, including the GLIA-CTN Advocacy Committee Chair and Co-Chair, and countless members of the Committee. In her role as GLIA-CTN Patient Advocacy Liaison, she is responsible for stakeholder engagement, meeting planning, newsletter preparation, and so much more.
Sarah graduated from West Chester University with a Master of Social Work in 2014, after she received her bachelor’s degree in the same discipline in 2006. She joined Children’s Hospital of Philadelphia’s Division of Neurology in 2014.
Connect with Sarah via LinkedIn at https://www.linkedin.com/in/stoneymswlsw.
Data Integration Core
Richard Aplenc, MD, PhD, MSCE
Children’s Hospital of Philadelphia
Dr. Richard Aplenc is an Associate Professor of Pediatrics in the Department of Pediatrics and a core faculty member of the Center for Pediatric Clinical Effectiveness at the Children’s Hospital of Philadelphia (CHOP). In addition to his medical training, which he completed at the University of Virginia, he received an MSCE in Clinical Epidemiology and Biostatistics, as well as a PhD in Epidemiology, at the University of Pennsylvania. He is the Chief Clinical Research Officer for the Children’s Hospital of Philadelphia, serves as Section Chief of Hematologic Malignancies, and is the Co-Director of the Clinical Trials Science Award at the Universityof Pennsylvania. Dr. Aplenc's research is focused on acquisition and integration of EMR data into clinicalepidemiology studies. His extensive prior experience in developing automated EMR data extraction tools andsenior leadership role in the CHOP Research Institute position him to facilitate work of the Data Integration Core.
Robert Grundmeier, MD
Children’s Hospital of Philadelphia
Robert Grundmeier, MD is a core faculty member at the Center for Pediatric Clinical Effectiveness (CPCE) at the Children's Hospital of Philadelphia, an attending physician and director of clinical informatics in the Department of Biomedical and Health Informatics, and an assistant professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.
Dr. Grundmeier’s informatics work focuses on the expansion of the electronic health record (EHR) interface to deliver interventions and decision support directly to healthcare providers within the clinical setting. He is board certified in clinical informatics and is considered a national expert in the repurposing of clinical data for secondary use in clinical and genomics Dr. Grundmeier is also a founding member of the Pediatric Research Consortium (PeRC), the primary care practice-based research network at The Children's Hospital of Philadelphia.
Justine Shults, PhD, MS
Children’s hospital of Philadelphia
Dr. Justine Shults, is a Professor of Biostatistics in the Department of Biostatistics,Epidemiology and Informatics at the University of Pennsylvania. She also serves as the faculty director of the Biostatistics and Data Management Core (BDMC) at the Children’s Hospital of Philadelphia.
Prior to coming to Penn, she was a faculty member at the Center for Pediatric Research, a joint program of Eastern Virginia Medical School and the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, where she was involved in clinical trials and community based intervention studies. Her methodological research focuses on the development of improved statistical approaches for the analysis of longitudinal data, with a more recent focus on discrete longitudinal data. She was funded by the National Science Foundation and was the principal investigator of a completed NIH funded R01 project that developed improved approaches for longitudinal analysis of data from diverse populations (LADP).
She is the lead author on a book that stems from the LADP project. She is currently the principal investigator of the NIH-funded Renal and Urologic Biostatistics Training Grant (T32DK060455) that aims to train graduate students in the Penn biostatistics program to be rigorous and independent academic investigators who use biostatistics to address issues in kidney and urology research.